通过Rasch模型与患者报告的结果进行共校准,为表现结果测量提供有意义的解释:多发性硬化症测量的例证。

IF 1.2 4区 医学 Q4 PHARMACOLOGY & PHARMACY
Antoine Regnault, Juliette Meunier, Anna Ciesluk, Wenting Cheng, Bing Zhu
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引用次数: 0

摘要

绩效结果(PerfO)测量基于患者在受控环境中执行的任务,因此很难对其进行有意义的解释。共同校准PerfO和相同目标概念的患者报告结果(PRO)测量值允许用PRO的项目内容解释PerfO。将Rasch模型应用于离散化的PerfO测量和PRO项目,允许在PRO度量中表示与PerfO测量相关的参数,以便将其与PRO响应相关联。我们将这种方法应用于两种用于多发性硬化症(MS)步行和手动能力的PerfO测量:定时25英尺步行(T25FW)和9孔Peg测试(9HPT)。为了确定这两个PerfO测量值的有意义的解释,他们使用来自5个全球MS临床试验的2043名受试者的数据,与两个密切相关概念的PRO测量值,MS步行量表-12项(MSWS-12)和ABILHAND共同校准。PerfO测量值和PRO指标之间的概率关系被用来表达对PRO项目的反应模式,作为PerfOs单位的函数。这个例子说明了解释PerfO测量的协同校准方法的前景,但也突出了与之相关的挑战,主要与PRO度量在目标概念的覆盖范围方面的质量有关。与PRO测量的共同校准也可以是解释数字传感器测量的适当解决方案,其意义也经常受到质疑。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Providing meaningful interpretation of performance outcome measures by co-calibration with patient-reported outcomes through the Rasch model: illustration with multiple sclerosis measures.

Performance outcome (PerfO) measures are based on tasks performed by patients in a controlled environment, making their meaningful interpretation challenging to establish. Co-calibrating PerfO and patient-reported outcome (PRO) measures of the same target concept allow for interpretation of the PerfO with the item content of the PRO. The Rasch model applied to the discretized PerfO measure together with the PRO items allows expressing parameters related to the PerfO measure in the PRO metric for it to be linked to the PRO responses. We applied this approach to two PerfO measures used in multiple sclerosis (MS) for walking and manual ability: the Timed 25-Foot Walk (T25FW) and the 9-Hole Peg Test (9HPT). To determine meaningful interpretation of these two PerfO measures, they were co-calibrated with two PRO measures of closely related concepts, the MS walking scale - 12 items (MSWS-12) and the ABILHAND, using the data of 2,043 subjects from five global clinical trials in MS. The probabilistic relationships between the PerfO measures and the PRO metrics were used to express the response pattern to the PRO items as a function of the unit of the PerfOs. This example illustrates the promises of the co-calibration approach for the interpretation of PerfO measures but also highlights the challenges associated with it, mostly related to the quality of the PRO metric in terms of coverage of the targeted concept. Co-calibration with PRO measures could also be an adequate solution for interpretation of digital sensor measures whose meaningfulness is also often questioned.

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来源期刊
Journal of Biopharmaceutical Statistics
Journal of Biopharmaceutical Statistics 医学-统计学与概率论
CiteScore
2.50
自引率
18.20%
发文量
71
审稿时长
6-12 weeks
期刊介绍: The Journal of Biopharmaceutical Statistics, a rapid publication journal, discusses quality applications of statistics in biopharmaceutical research and development. Now publishing six times per year, it includes expositions of statistical methodology with immediate applicability to biopharmaceutical research in the form of full-length and short manuscripts, review articles, selected/invited conference papers, short articles, and letters to the editor. Addressing timely and provocative topics important to the biostatistical profession, the journal covers: Drug, device, and biological research and development; Drug screening and drug design; Assessment of pharmacological activity; Pharmaceutical formulation and scale-up; Preclinical safety assessment; Bioavailability, bioequivalence, and pharmacokinetics; Phase, I, II, and III clinical development including complex innovative designs; Premarket approval assessment of clinical safety; Postmarketing surveillance; Big data and artificial intelligence and applications.
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